50 patents
Page 3 of 3
Utility
Nucleic Acid Constructs and Methods of Use
26 Aug 20
Nucleic acid constructs that allow insertion and/or expression of a sequence of interest, such as a transgene, are provided.
John Finn, Hon-Ren Huang
Filed: 17 Oct 19
Utility
Compositions and Methods for Treating ALPHA-1 Antitrypsin Deficiency
26 Aug 20
Compositions and methods for expressing alpha 1 antitrypsin (AAT) in a host cell are provided.
John Finn, Hon-Ren Huang, Anthony Forget, Xin Xie
Filed: 17 Oct 19
Utility
Compositions and Methods for Transgene Expression from an Albumin Locus
26 Aug 20
Methods for editing, e.g., introducing a heterologous transgene, within the human albumin gene (e.g., at intron 1) are provided.
John Finn, Hon-Ren Huang, Moitri Roy, KehDih Lai, Rachel Sattler, Christos Kyratsous, Cheng Wang
Filed: 17 Oct 19
Utility
Compositions and Methods for TTR Gene Editing and Treating ATTR Amyloidosis
5 Aug 20
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided.
Arti Mahendra Prakash Kanjolia, Shobu Odate, Jessica Lynn Seitzer, Reynald Michael Lescarbeau, Walter Strapps
Filed: 23 Mar 20
Utility
Stabilized Nucleic Acids Encoding Messenger Ribonucleic Acid (mRNA)
3 Jun 20
This disclosure relates to the field of poly-adenylated (poly-A) tails.
Christian Dombrowski
Filed: 13 Feb 20
Utility
Methods for the Treatment of Disease with Gene Editing Systems
6 May 20
Provided herein are methods of selectively treating a patient with a gene editing system on the basis of ascertaining the presence of a target sequence, at a target locus, that is fully complementary to a targeting domain of said gene editing system and/or on the basis of ascertaining the absence of a target sequence, at a locus other than the target locus, that is fully complementary to a targeting domain of said gene editing system.
Nicole RENAUD, Xiaojun ZHAO
Filed: 27 Jun 18
Utility
Methods and Compositions for Ocular Cell Therapy
29 Apr 20
The present invention provides ocular cells, genetically modified by a CRISPR system targeting the expression of B2M for ocular cell therapy.
Frada BERENSHTEYN, Bo HAN, Xueshi HAO, Jessica HEYDER, Timothy Z. HOFFMAN, Qihui JIN, Arnaud LACOSTE, Jun LIU, Yahu LIU, Tingting MO, Bradley Andrew MURRAY, Daniel Joseph O'CONNELL, Jianfeng PAN, Yun Feng XIE, Shanshan YAN, Yefen ZOU
Filed: 24 Oct 19
Utility
Compositions and Methods for the Treatment of Hemoglobinopathies
1 Apr 20
The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.
Craig Stephen MICKANIN, Christian SCHMEDT, Jennifer SNEAD, Susan Stevenson, Yi YANG
Filed: 4 Feb 18
Utility
Modified Guide RNAs
16 Oct 19
This disclosure relates to modified single and dual guide RNAs having improved in vitro and in vivo activity in gene editing methods.
Amy Madison Rhoden Smith, David V. Morrissey, Walter Strapps
Filed: 6 Jun 19
Utility
Compositions and Methods for Treating Alpha-1 Antitrypsin Deficiency
16 Oct 19
Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided.
Shobu Odate, Walter Strapps, Reynald Michael Lescarbeau
Filed: 19 Jun 19